Gaucher DiseaseApsNCT04050137
Therapeutic Exercise to Treat Neuropathic Pain
A study testing Therapeutic Exercise in people with Gaucher Disease and Fabry Disease, run by Universidad de Zaragoza at 1 site in Spain.
Trial summary
A quasi-experimental, prospective clinical trial with pre and post intervention measurements, whose porpuose is assess the efficacy of a therapeutic exercise protocol to treat neuropathic pain in Fabry Disease. Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Gaucher Disease, Fabry Disease
- What is being tested?
- Therapeutic Exercise
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteSpain
- Study dates
- 2 May 2019to 14 Dec 2020
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Female or male patients between 18 years and 65 years.
- Patients diagnosed by a doctor specializing in Fabry disease with FabryScan.
- Fabry's disease must have a stable evolution, with a disease severity scoring system (DS3) that must be less than 8 points per year in the last 2-3 years.
- Presence of neuropathic pain with a score greater than or equal to 4 in the "Douleur neuropathique (DN4)" questionnaire.
Who cannot join
- Patients with an acute cardiovascular disease or with a heart or kidney transplant.
- Subjects with acute orthopedic problems that limit their participation in the study.
- Subjects with cognitive impairments that prevent them from filling in the questionnaires or understanding the exercises to be performed.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Therapeutic Exercise to Treat Neuropathic Pain in Patients With Chronic Lisosomal Injuries: Learning and Service Project
A quasi-experimental, prospective clinical trial with pre and post intervention measurements, whose porpuose is assess the efficacy of a therapeutic exercise protocol to treat neuropathic pain in Fabry Disease.
Read the detailed description
There take part in this study women or men aged between 18 and 65 years, with a diagnosys by a physician specializing in Fabry disease with FabryScan. Patients must present a stable evolution, with a scoring system of disease severity (DS3) that must be less than 8 points per year in the last 2-3 years and they have to presence of neuropathic pain with a score greater than or equal to 4 in the questionnaire "Douleur neuropathique (DN4)".
- Conditions
- Gaucher Disease, Fabry Disease
- Being tested
- Therapeutic Exercise
- Keywords
- Gaucher Disease, Exercise, Terapheutic
- Medical subject headings
- Gaucher Disease, Fabry Disease, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Therapeutic ExerciseOther
Patients who meet the inclusion criteria and who want to carry out the workshops, fill out an informed consent explaining the procedure to be followed and accepting the cessation of data to carry out the study.
Patients will be divided into 2 working groups (approximately 10 patients in total, in a Gaucher disease group and in another Fabry disease). The workshops will be held at the foundation twice a month in sessions of 45 minutes of work for 3 months (May, June, July 2019). In these sessions the physiotherapists will show the new exercises to the patients. The exercises that are taught in that session should be done at home 3 times a week. In addition, a document will be delivered with the exercises to be carried out and the frequency and intensity of execution. The workshops will be guided at all times by a physiotherapist specialized in therapeutic exercise and EDL.
| Study group | Role | What they receive |
|---|---|---|
| Exercise group | Experimental | Exercise programme 3 times/week. Receives: Other: Therapeutic Exercise |
What is being measured
The primary outcome is the main question the study is designed to answer.
Neurophatic pain
DN4. This tool allows to estimate the probability of neuropathic pain of the patient in a valid and reliable way. It consists of 4 questions with 10 items to fill out. The researcher directly asks the patient and it is the investigator who completes the questionnaire. To each item, you must provide a "yes" or "no" answer; the "yes" is worth 1 point and the "no" is worth 0 point. At the end of the questionnaire, the investigator counts the patient's score on 10. If the patient's score is equal to or greater than 4 out of 10, the test is positive.
Measured: 8 weeks
Secondary outcomes (9)
Functionality
Arm Functionality DASH. The DASH questionnaire is a tool for assessing and measuring the functionality of the upper limb that the patient perceives during daily activities. This questionnaire is used in several studies with patients with disabilities in the hand, elbow and shoulder. It is valid, reliable and capable of detecting changes.
Measured: 8 weeks
Legs' strenght
Test of rising and sitting of the chair during 30 seconds as many times as possible.
Measured: 8 weeks
Strenght of the extensor muscless of the trunk
BST. Biering-Sorensen test.The test is to keep the trunk in prone position as long as possible to assess the resistance of the trunk extensor musculature.
Measured: 8 weeks
Strenght of the inclination muscless of the trunk
TSB. Test Side-Bridge assesses the resistance of the inclination muscles or lateral flexors of the trunk. In it the participants are placed in lateral recumbency on their dominant side in a mat. In this position, the participants support themselves with the elbow and the forearm of their dominant side and raise the pelvis until the trunk is aligned with the lower extremities. The test consists of maintaining the referred position as long as possible.
Measured: 8 weeks
Strenght of the flexor muscless of the trunk
ITo. To assess the resistance of the flexor muscles of the trunk, the participants are placed supine with the hips and knees bent at 90º. The participants flex the upper part of the trunk until they touch the thighs with the elbows, keeping the cervical spine in a neutral position.
Measured: 8 weeks
Visual Analogue Scale
VAS. The analog visual pain scale is a tool that is used to assess the pain perceived by the patient in a valid and reliable way.
It is an instrument for measuring the intensity of pain that represents a horizontal line of 10 cm with a score of 0 to 10, where 0 represents non-pain and 10 unbearable pain.
The patient should place a vertical line on the horizontal line of the scale where he places his pain intensity.
Measured: 8 weeks
Hospital, anxiety and depression questionnaire
HADS. This questionnaire allows to evaluate the psychosocial factors of the patient, through 14 questions with two subscales of 7 questions (one subscale on anxiety and the other on depression); The patient must answer one of the 4 items of each question without much reflection to have a real answer of how he was during the past week. The intensity or frequency of symptoms is evaluated with a Likert scale ranging from 0 to 3. In each subscale (anxiety and depression) the points are added and interpreted as such: 0-7 points: normal; 8-10 points: doubtful and higher than 11: clinical problem. This tool is validly and reliably used in clinical practice.
Measured: 8 weeks
SF-36
This questionnaire assesses the quality of life perceived by the patient. It is widely used in clinical practice and is approved as valid and reliable. There are 36 questions with 2,3,5 or 6 items in each question and with an assessment between 0 and 100. The questions assess physical function, function limitations due to physical health problems, body aches, general health, vitality, social functioning and emotional and mental health problems
Measured: 8 weeks
Semmes Weinstein monofilament test
The Semmes Weinstein monofilament test is a tool that evaluates tactile and pressure sensitivity in a valid and reliable way. A nylon filament attached to a handle is used which, when folded, applies a constant pressure of 10g. The investigator applies the monofilament perpendicular to the skin with increased pressure until the monofilament bends and without supporting it for more than 1 or 2 seconds.
The test will be performed on the sole of the foot in 4 points: the first toe, the base of the first, third and fifth metatarsals.
When the patient notices something, he puts a 1 and a 0 if he does not notice something. The sensitivity index is obtained with the sum of the values and is considered a sensitive patient only when it has a score of 8/8.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2019
- Study start2 May 2019
- Primary completion1 Dec 2020
- Study completion14 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Universidad de Zaragoza
- Sponsor type
- University, hospital or other
- Collaborators
- fundación española para el estudio y tratamiento de la Enfermedad de Gaucher y otras lisosomales, Fundación San Valero, Fundación Ibercaja
- Responsible party
- Principal investigator, Maria Pilar López Royo, Universidad de Zaragoza
- Investigators
- Royo, Principal investigator, Universidad San Jorge
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04050137
- Sponsor's protocol ID
- PI19/205
- Acronym
- Aps
- Status last verified
- Dec 2020
- First submitted
- 23 Apr 2019
- Last updated
- 16 Dec 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Therapeutic Exercise to Treat Neuropathic Pain in Patients With Chronic Lisosomal Injuries: Learning and Service Project
- ClinicalTrials.gov ID
- NCT04050137
- Acronym / sponsor ID
- Aps / PI19/205
- Conditions
- Gaucher Disease, Fabry Disease
- Keywords
- Gaucher Disease, Exercise, Terapheutic
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Universidad de ZaragozaUniversity, hospital or other
- Collaborators
- fundación española para el estudio y tratamiento de la Enfermedad de Gaucher y otras lisosomales, Fundación San Valero, Fundación Ibercaja
- Study start
- 2 May 2019 (actual)
- Primary completion
- 1 Dec 2020 (actual)
- Study completion
- 14 Dec 2020 (actual)
- First posted
- 8 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A quasi-experimental, prospective clinical trial with pre and post intervention measurements, whose porpuose is assess the efficacy of a therapeutic exercise protocol to treat neuropathic pain in Fabry Disease.
Detailed description
There take part in this study women or men aged between 18 and 65 years, with a diagnosys by a physician specializing in Fabry disease with FabryScan. Patients must present a stable evolution, with a scoring system of disease severity (DS3) that must be less than 8 points per year in the last 2-3 years and they have to presence of neuropathic pain with a score greater than or equal to 4 in the questionnaire "Douleur neuropathique (DN4)".
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Exercise group | Experimental | Other: Therapeutic ExerciseExercise programme 3 times/week. |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Female or male patients between 18 years and 65 years.
- Patients diagnosed by a doctor specializing in Fabry disease with FabryScan.
- Fabry's disease must have a stable evolution, with a disease severity scoring system (DS3) that must be less than 8 points per year in the last 2-3 years.
- Presence of neuropathic pain with a score greater than or equal to 4 in the "Douleur neuropathique (DN4)" questionnaire.
Key exclusion criteria (3 listed)
- Patients with an acute cardiovascular disease or with a heart or kidney transplant.
- Subjects with acute orthopedic problems that limit their participation in the study.
- Subjects with cognitive impairments that prevent them from filling in the questionnaires or understanding the exercises to be performed.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Therapeutic ExercisePatients who meet the inclusion criteria and who want to carry out the workshops, fill out an informed consent explaining the procedure to be followed and accepting the cessation of data to carry out the study. Patients will be divided into 2 working groups (approximately 10 patients in total, in a Gaucher disease group and in another Fabry disease). The workshops will be held at the foundation twice a month in sessions of 45 minutes of work for 3 months (May, June, July 2019). In these sessions the physiotherapists will show the new exercises to the patients. The exercises that are taught in that session should be done at home 3 times a week. In addition, a document will be delivered with the exercises to be carried out and the frequency and intensity of execution. The workshops will be guided at all times by a physiotherapist specialized in therapeutic exercise and EDL. | Other | Exercise group |
Primary outcome
Neurophatic pain
Time frame: 8 weeks
DN4. This tool allows to estimate the probability of neuropathic pain of the patient in a valid and reliable way. It consists of 4 questions with 10 items to fill out. The researcher directly asks the patient and it is the investigator who completes the questionnaire. To each item, you must provide a "yes" or "no" answer; the "yes" is worth 1 point and the "no" is worth 0 point. At the end of the questionnaire, the investigator counts the patient's score on 10. If the patient's score is equal to or greater than 4 out of 10, the test is positive.
9 secondary outcomes
Functionality
Time frame: 8 weeks
Legs' strenght
Time frame: 8 weeks
Strenght of the extensor muscless of the trunk
Time frame: 8 weeks
Strenght of the inclination muscless of the trunk
Time frame: 8 weeks
Strenght of the flexor muscless of the trunk
Time frame: 8 weeks
Visual Analogue Scale
Time frame: 8 weeks
Hospital, anxiety and depression questionnaire
Time frame: 8 weeks
SF-36
Time frame: 8 weeks
Semmes Weinstein monofilament test
Time frame: 8 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Dec 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Royo principal investigator
Universidad San Jorge
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Therapeutic Exercise in people with Gaucher Disease and Fabry Disease, run by Universidad de Zaragoza at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Neurophatic pain (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Dec 2020.